• Primary objective is to determine efficacy of chronic treatment with arimoclomol compared to placebo over 76 weeks

  • Results expected in H1 2021

Copenhagen, August 10, 2018 Orphazyme A/S, a biopharmaceutical company dedicated to developing treatments for patients living with rare diseases, today announced that the first patient has received the first dose in its Phase III clinical trial of arimoclomol for Amyotrophic Lateral Sclerosis (ALS).

Thomas Blaettler, Chief Medical Officer, said: “The enrollment of the first patient in our Phase III trial for ALS represents a major milestone for the company. Arimoclomol has shown substantial promise in previous trials and has a favorable safety and tolerability profile. This is a truly devastating disease and we will work to expeditiously advance the Phase III trial with the prospect of making a new therapeutic option available to the patients in the shortest possible timeframe”.

Michael Benatar, MD, PhD, University of Miami, Principal Investigator of a prior Phase II trial of arimoclomol in SOD1 ALS and Lead International Coordinating Investigator, said: “I am delighted to see arimoclomol advance into Phase III testing and that the eligible population has been broadened to include all patients with ALS. Based on the mechanism of action of the drug and our understanding of the underlying biology of ALS, there is good reason to believe that all patients with ALS might benefit from this therapeutic approach”.

Lucie Bruijn, PhD, MBA, Chief Scientist at the ALS Association, said: “On behalf of the patients struck by this devastating disease, I am delighted to see this Phase III trial up and running. The ALS Association invested in the early clinical trials and are pleased to see this moving forward to a Phase III study and to seeing the potential of arimoclomol unfold”.

The randomized and placebo-controlled Phase III trial in ALS is being conducted in North America and Europe and will enroll 231 patients, to be randomized in a 2:1 ratio receiving either arimoclomol or placebo for up to 76 weeks. Patients completing the trial will be offered participation in an open-label extension trial.

The primary objective of the present trial is to determine the efficacy of chronic treatment with arimoclomol compared to placebo over 76 weeks in subjects with ALS as assessed with a combined assessment of function and survival (CAFS). Important secondary endpoints include survival, change in ALSFRS-R, and slow vital capacity (SVC).

A total of 30 sites are planned in the US, Canada and Europe. 

Results from the Phase III trial are expected in H1 2021.